An expensive new medicine is finally available for a rare disease – it's been fighting for two years to get it

When Annie Wilson was just six months old, she was diagnosed with spinal muscular atrophy, a rare disease that causes progressive muscle weakness and impaired movement – and told she would not live past age. her condition was approved in Biogen Inc.'s

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and Ionis Pharmaceuticals Inc.'s

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Spinraza. Spinraza has attracted attention for being innovative, up to $ 750,000 in the first year, and $ 375,000 a year afterwards. By the time of approval, Wilson was having trouble breathing, talking and even driving her wheelchair, and was excited by the prospect of the new treatment. But Kaiser Permanente, Wilson said MarketWatch, said she was too weak and should come back later. Wilson has challenged Kaiser's decision through a Medicare appeals process. Even so, the two-year-long battle has been exhausting, Wilson told MarketWatch in an email. She suspects the stress is affecting her health, and has even considered giving up. "I did not want to die for a drug that will stop the progression of my disease," Wilson said. "I feel like, by denying someone, it's just like," "I was not getting spinraza, I'm not expecting a miracle," she said. "Stopping the progression of my disease is what I am aiming for. Preserving the little movement I am most important to my quality of life. "

Annie Wilson, a 36-year-old from Alameda, California, was first diagnosed with the rare disease spinal muscular atrophy when she was six months old. She has been struggling with Kaiser Permanente over the last two years to get access to an expensive therapy that could halt the disease's progression. See more: Extremely expensive rare-disease drug could not be used in the treatment of patients with schizophrenia. Dr. Sameer Awsare, Associate Executive Director of The Permanent Medical Group, said in a statement. "Spinach is a major contributor to spinal muscular atrophy, and is more likely to be associated with spinal muscular atrophy. But Wilson's experience speaks to the tangle of complications that can keep promising therapies away from patients. Related: About 10,000 to 25,000 individuals in the US are thought to have SMA, which is an inherited disorder, according to the SMA Foundation. Most of the total population, or about 60%, are adults. Biogen's most closely watched product – and could be a blockbuster Spinraza was approved in late 2016 for both children and adults, but some health insurers – including Kaiser – balked at wrapping adults, since clinical trials that got Spinraza approved only tested it in children. More adults with SMA are now being treated with Spinraza, including by Kaiser, Wilson's patient advocate, Joahn Ginsberg said. "They have to treat her, and they need to pay for that treatment, because that's what she pays for in Medicare every month," Ginsberg said. UnitedHealthcare guidelines do vary, though, and major health insurers

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and Cigna Corp.

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have forced the use of ventilators. In particular, SMA is a key factor influencing restrictions on Spinraza access. See: ALS patient group unhappy with how $ 115 million raised by the Ice Bucket Challenge is being spent in the US Nonetheless, those who rely on ventilators. Stanford University Hospital, for example, had treated 32 adults as of mid-2018, 22 of which were not able to be ventilated, and neuromuscular team feels Wilson's doctor at Kaiser earlier this year. The Stanford team also recommends that "treatment of [Annie] with Spinraza is reasonable and would be beneficial in halting progression of disease and possibly improving existing function, "according to the letter. Biogen has been estimated to be 15% of adult patients, compared with approximately half of all infants and pediatric patients. A spokesperson told MarketWatch that Spinraza has "demonstrated significant and clinically meaningful efficacy … across a broad range of SMA populations." There are a large number of untreated adult patients that we believe could benefit from Spinraza, "Jeffrey Capello, Biogen's chief financial officer , said the October conference call. But questions about price tag, patient and side effects are likely to linger, and for Wilson, time is everything. "Is it going to miraculously cure her SMA? No, "Ginsberg said. "But do we think it can help her? Yes. "

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